Table 1.
All approved gene therapies, including genetically modified cell therapies, as of June 2023
| Product name | Generic name | Originator company | Modality | Disease(s) | Year first approved | Locations approved |
|---|---|---|---|---|---|---|
| Vyjuvek | Beremagene geperpavec | Krystal Biotech | HSV-1 gene therapy | Epidermolysis bullosa | 2023 | USA |
| Adstiladrin | Nadofaragene firadenovec | Merck | Adenoviral gene therapy | Bladder cancer | 2022 | USA |
| Hemgenix | Etranacogene dezaparvovec | uniQure | AAV5 gene therapy | Hemophilia B | 2022 | USA, EU, UK |
| Roctavian | Valoctocogene roxaparvovec | BioMarin | AAV5 gene therapy | Hemophilia A | 2022 | EU, UK |
| Upstaza | Eladocagene exuparvovec | PTC Therapeutics | AAV2 gene therapy | Aromatic l-amino acid decarboxylase deficiency | 2022 | EU, UK |
| Carvykti | Ciltacabtagene autoleucel | Legend Biotech | CAR-T | Myeloma | 2022 | USA, EU, UK, Japan |
| Skysona | Elivaldogene autotemcel | Bluebird Bio | Genetically modified autologous CD34+ hematopoietic stem cells | Adrenoleukodystrophy | 2021a | USA |
| Delytact | Teserpaturev | Daiichi Sankyo | Oncolytic virus | Cancer, brain | 2021 | Japan |
| Benoda | Relmacabtagene autoleucel | JW Therapeutics | CAR-T | Cancer, lymphoma, B cell, diffuse large; cancer, lymphoma, follicular | 2021 | China |
EU, European Union; UK, United Kingdom.
a
Approved in the EU, but then was withdrawn.